The NIH Loosens Grip on Gene Therapy Trials

The agency proposes ceding its scrutiny of these studies to the FDA.

Aug 16, 2018

The directors of the National Institutes of Health and the US Food and Drug Administration propose limiting the role of the NIH in assessing proposals for gene therapy experiments, the two explain in a commentary in the New England Journal of Medicine published yesterday (August 15). The NIH’s Francis Collins and FDA’s Scott Gottleib say that the “oversight burden” for gene therapy trials is duplicated by having both agencies involved, and FDA can handle the task on its own.

“We have mechanisms in place to protect patients,” Jeffrey Kahn, director of the Bioethics Institute at Johns Hopkins University, tells the Associated Press. “It doesn’t need to be treated as a special case of clinical research any longer.”

The specific plan, which will be posted to the Federal Register for public comment tomorrow, is to redirect a special NIH panel called the Recombinant DNA Advisory Committee (RAC) away from scrutinizing gene therapy proposals to focus on newer biotechnologies. “In the view of the senior leaders of the FDA and the NIH, there is no longer sufficient evidence to claim that the risks of gene therapy are entirely unique and unpredictable—or that the field still requires special oversight that falls outside our existing framework for ensuring safety,” Collins and Gottlieb write.

RAC was established in 1974 and has evolved over time. In recent years, the panel began to ease its oversight of gene therapy experiments, in accordance with recommendations from the Institute of Medicine. In 2016, RAC decided to only review trials with “exceptional issues or concerns,” a number totaling just three out of 275 protocols, the directors say.

Not everyone is a fan of the proposed oversight shift. “This is not the right time to be making any moves based on the idea that we know what the risks are,” Stanford University bioethicist Mildred Cho, a RAC panel member, tells the Associated Press. She contends scientists don’t understand fully how these complex interventions work—and why they sometimes don’t.